TW202323524A - Ｈｂｂ—調節組合物及方法 - Google Patents

Ｈｂｂ—調節組合物及方法

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Publication number
TW202323524A
TW202323524A TW111134206A TW111134206A TW202323524A TW 202323524 A TW202323524 A TW 202323524A TW 111134206 A TW111134206 A TW 111134206A TW 111134206 A TW111134206 A TW 111134206A TW 202323524 A TW202323524 A TW 202323524A
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TW
Taiwan
Prior art keywords
sequence
domain
grna
template
template rna
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TW111134206A
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English (en)
Chinese (zh)
Inventor
羅伯特 查爾斯 奧茲胡勒
安 海倫 巴斯摩爾
丹尼爾 雷蒙 朱
拉莫西諾 賽西莉亞 喬凡娜 賽爾維雅 寇特
金奎植
蘭迪 蜜雪爾 寇特拉爾
葛雷哥萊 大衛 麥克艾力斯特
安娜亞 雷
納薩尼珥 羅奎特
卡洛斯 桑切斯
巴雷特 伊森 史丹伯格
威廉 愛德華 薩洛蒙
羅伯特 詹姆士 西圖瑞克
威廉 奎比斯
路西安諾 亨瑞克 阿波尼
展 王
傅豔芳
丹尼爾 金 亞伯納西
麥可 克里斯多福 赫姆斯
Original Assignee
美商旗艦先鋒創新有限責任公司
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Application filed by 美商旗艦先鋒創新有限責任公司 filed Critical 美商旗艦先鋒創新有限責任公司
Publication of TW202323524A publication Critical patent/TW202323524A/zh

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/13011—Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hematology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
TW111134206A 2021-09-08 2022-09-08 Ｈｂｂ—調節組合物及方法 TW202323524A (zh)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US202163241994P 2021-09-08 2021-09-08
US63/241,994 2021-09-08
US202163250143P 2021-09-29 2021-09-29
US63/250,143 2021-09-29
US202263303900P 2022-01-27 2022-01-27
US63/303,900 2022-01-27
PCT/US2022/076063 WO2023039440A2 (en) 2021-09-08 2022-09-07 Hbb-modulating compositions and methods
WOPCT/US22/76063 2022-09-07

Publications (1)

Publication Number Publication Date
TW202323524A true TW202323524A (zh) 2023-06-16

Family

ID=85506910

Family Applications (1)

Application Number Title Priority Date Filing Date
TW111134206A TW202323524A (zh) 2021-09-08 2022-09-08 Ｈｂｂ—調節組合物及方法

Country Status (7)

Country Link
US (1) US20240252682A1 (https=)
EP (1) EP4399307A4 (https=)
JP (1) JP2024533313A (https=)
AU (1) AU2022342169A1 (https=)
CA (1) CA3231679A1 (https=)
TW (1) TW202323524A (https=)
WO (1) WO2023039440A2 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
WO2024197103A2 (en) * 2023-03-21 2024-09-26 Tessera Therapeutics, Inc. Modified template guide rna molecules
WO2024206125A1 (en) * 2023-03-24 2024-10-03 The Broad Institute, Inc. Use of prime editing for treating sickle cell disease
WO2025059607A1 (en) * 2023-09-13 2025-03-20 Tessera Therapeutics, Inc. Lipid nanoparticles for delivery of therapeutic payloads to cells
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2017252023B2 (en) * 2016-04-18 2024-05-02 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
KR20190039703A (ko) * 2016-07-05 2019-04-15 더 존스 홉킨스 유니버시티 Crispr/cas9-기반 조성물 및 망막 변성을 치료하기 위한 방법
WO2020191233A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
AU2020344547A1 (en) * 2019-09-09 2022-03-24 Beam Therapeutics Inc. Novel nucleobase editors and methods of using same
US12435330B2 (en) * 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA

Also Published As

Publication number Publication date
WO2023039440A9 (en) 2023-07-06
JP2024533313A (ja) 2024-09-12
CA3231679A1 (en) 2023-03-16
US20240252682A1 (en) 2024-08-01
EP4399307A2 (en) 2024-07-17
AU2022342169A1 (en) 2024-03-28
EP4399307A4 (en) 2026-01-21
WO2023039440A2 (en) 2023-03-16
WO2023039440A3 (en) 2023-05-19

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